CRISPR Gene Therapy Could Advance Using "Jumpers" – Axios

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Two major teams of scientists recently announced that transposons – or "jumping genes" – could improve the accuracy of CRISPR gene editing.

Why it's important: While this research is still in its infancy, while both teams have tested their techniques on bacterial cells, experts say that this technique could allow more precise insertion of modified genes into genomes, which could resolve problems with current CRISPR systems that may result in non-targeted editing. and random deletions or even cancer.

Context: The transposons jump randomly from one site to another, inserting genetic information as and when required, using enzymes called transposases.

  • CRISPR tools currently use enzymes such as Cas9 and Cas13 to cut and remove some of the genetic code, relying on the fact that the cell uses its repair function to pick up the cut strands. This process sometimes introduces its own problems.
  • By combining the CRISPR tool with these transposons, which make it easy to introduce a large number of genes into cells, the researchers hope to merge the best of both worlds, says Ilya Finkelstein, assistant professor at the University of California. University of Texas at Austin, which part of one or the other study.

Details: The two studies essentially combine CRISPR with different transposons and, in so doing, reveal details of how natural CRISPR systems have evolved and why there are a large number of systems.

Study 1. CAST – This system, which corresponds to the transposase associated with CRISPR, has been published in Science last week by a team led by the Broad Institute of MIT and Harvard University.

  • "This makes it possible to fill a void that we could not tackle before, to be able to insert DNA into the genome," says author of the study, Feng Zhang, about the possibility to insert large genomes in a targeted way.
  • They found that the system, which uses a protein called Cas12K and transposed by Tn-7, produced the desired editing up to 80% of the time, which is superior to the current CRISPR tools (about 20%) . But, there were still a number of insertions off target.

Study 2. Cascade – This system was developed primarily by Columbia University and was published in Nature Wednesday.

  • Sam Sternberg, author of the study, explains to Axios: "Our discovery of RN-guided integrases offers a new approach, with the same programmability and ease of use as CRISPR – Cas9, but without the undesirable effects associated with DNA breaks. "
  • "Our system provides both scissors and glue, eliminating the requirements for DNA breaks and host factors," says Sternberg.
  • It was found that this produced the desired editing up to 60% of the time, but had fewer off-target insertions than the CAST system, Sternberg adds.

And after: The researchers plan to test the systems in the cells of plants and animals, and then see if it can be used to treat certain human diseases.

  • "There will soon be a transposon / CRISPR patent explosion," predicts Finkelstein.

MeanwhilePharmaceutical giant GlaxoSmithKline on Thursday announced a collaboration called LGR with another CRISPR scientist, Jennifer Doudna and UC Berkeley.

  • Doudna, who spoke at a press conference, said it was the "perfect marriage" to take advantage of the various forces of the groups.
  • She added that the combination of CRISPR technology to probe the underlying genomics of cells and that machine learning and drug development technology would lead to new gene therapies.

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